I stand here to stick up for patients in Scotland who have no voice: the patients with breast cancer or cystic fibrosis who have been denied access to vital, life-prolonging medicines due to bureaucracy. They have been failed by a system that has placed a value on their life and decided that the price tag is too high. Mums or dads, a son or daughter or a brother or sister—they are people who need this Parliament to stand up for them and make their voice heard in the hope that the Scottish Government listens and acts.
Many of the patients we are speaking of do not have much time
. It is too late for many of them to benefit from the medicines. What time they do have, they are using to campaign so that others do not find themselves in the same position of knowing that they have a terminal illness and that there are drugs available that will allow them to spend more time with their families: time that they will not get without the drugs that they need. One of those drugs is Perjeta, a breast cancer drug that prolongs someone’s life for 16 months, which is 16 months more with their loved ones. Breast Cancer Now deserves our thanks for leading that campaign, helped by the bravery of patients. Today, we are their voice.
Last week, Jon Ashworth, the shadow Secretary of State for Health and Social Care in England, and I met Breast Cancer Now and campaigners in Edinburgh. It is completely unacceptable that Jon Ashworth’s constituents in Leicester would be given access to Perjeta to prolong their lives but that my constituents in Glasgow are being denied that access. Women in England, Wales and Northern Ireland can get Perjeta on the national health service as a matter of course, but women in Scotland cannot get Perjeta. The drug has been rejected three times by the Scottish Medicines Consortium as it is not considered cost effective, despite it being recognised as clinically effective. I ask again: what cost life?
The other drug referred to in our motion is the life-prolonging cystic fibrosis drug Orkambi. It slows the decline in lung function—the main cause of death in cystic fibrosis—by 42 per cent. Orkambi also cuts the number of infections that require hospitalisation by more than 60 per cent. As we heard in a debate last week, the longer the delay in approving access to Orkambi, the greater the decline in lung function for cystic fibrosis sufferers.
That is why the matter has to be treated urgently. Professor Gordon MacGregor, a cystic fibrosis consultant, spoke about his anger that he has in his cabinet the drugs to prolong his patient’s life but he does not have the permission to prescribe them. On some occasions, the pharmaceutical company has even given the drug for free on compassionate grounds, but the clinician still cannot prescribe it, due to bureaucracy.
I thank the Cystic Fibrosis Trust, the patients and the campaigners. However, it should not take individuals’ courage in coming forward and sharing their very personal stories on the front page of a national newspaper, or indeed some individuals, as in the example of Anne Maclean-Chang, crowdfunding on Facebook, for the Scottish Government to take action and for them to get the vital drugs that they need.
That is why the Government must implement in full and without delay the recommendations in the Montgomery review, which was published in December 2016. In particular, the Government must deliver the ability to negotiate on price during the approval process. It makes sense that, if the drug has been accepted as clinically effective but discussions on cost effectiveness are on-going, the Scottish Medicines Consortium and NHS National Services Scotland should be able to negotiate with the pharmaceutical companies without asking them to reapply in a process that can take months and months.
It appears that the Government will support our motion, and that is welcome. However, for the cabinet secretary to write to me confirming which recommendations will be taken forward by the end of 2018—which is welcome—just shortly before this debate; for her to write to Jackie Baillie and Alex Neil just today about issues that they have been campaigning on; and for her to write to the chair of the Health and Sport Committee on these vital issues just yesterday only helps to emphasise that it should not take campaigners on a front page and us having to lodge motions in this Parliament to get action from the minister and the Government so that people can get access to life-saving or life-prolonging drugs.
As I said, I welcome the fact that the minister has indicated that she will support our motion, but can she confirm that the ability to negotiate on price will begin now, as the motion calls for, and not at some undefined point in 2018? Every single day that is lost in that process is a day when people are denied access to medicines, so now has to mean now. Will the cabinet secretary confirm to patients and clinicians when she expects Perjeta and Orkambi to be available to them?
The Government’s amendment states that the decisions on the new individual patient treatment request process—the peer-approved clinical system 2 process—will not be based on cost. Although that is welcome in words, health boards are in practice facing budgetary pressures that mean that they will have to cut up to £1 billion over the next four years. Therefore, will the Government guarantee two things? First, will it guarantee that the new PACS 2 process will be faster in delivering access to medicines than the previous process was? Secondly, will it guarantee that additional funds will be made available to health boards so that they can approve access to vital medicines for individual patients without knock-on pressures on existing services?
I go further and ask that the Government considers a portfolio deal on cystic fibrosis medicines. Last week, the Minister for Public Health and Sport, Aileen Campbell, said that that was not possible, as the Government could not spend money on drugs that have not been approved. I say to her that it starts to cost money only when the drug has been approved and is being prescribed by clinicians.
I want to end with the brave words of the campaigners. Breast cancer patient Jen Hardy said about Perjeta:
“Someone an hour and a half down the road can get it but I can’t. We shouldn’t have to think about cost because people in England and Wales don’t need to ... It is terrible not only for me but my family as well.”
“I think about what 16 months would mean for me. It’s a graduation, a wedding, knowing your kids are doing ok. We need this drug now to stop women dying earlier than they should.”
Jen Hardy will not get Perjeta, but she is campaigning for others to get it.
Kelli Gallacher, who is 24 and has cystic fibrosis, has been told not to expect to live beyond 31. Her letter to the First Minister says:
“I don’t have time to wait. These drugs are available in other countries and, to me, it feels like they have been put on a shelf just out of my reach. I know they are there but I can’t get to them.
More people with” cystic fibrosis
“will die unless something is done. We need these drugs now. Please don’t let us die.”
For patients with breast cancer or cystic fibrosis, every day matters. I ask members to please support our motion and make today matter.
That the Parliament calls on the Scottish Government, as a matter of urgency, to bring NHS National Services Scotland, the Scottish Medicines Consortium and the relevant pharmaceutical companies together to deliver access to the life-prolonging medicines, Perjeta and Orkambi, for patients in Scotland who need them; notes the commitment that was made by the Scottish Government in December 2016, following the Review of Access to New Medicines (the Montgomery Review), to improve ways of negotiating with drug companies on the cost of medicines, and calls for a new system of negotiation to be implemented now.
In recent years, the Parliament has driven significant change in access to new medicines, for which the system is—rightly—independent of politicians. Reforms to the availability of new medicines for rare, very rare and end-of-life conditions mean that the Scottish Medicines Consortium now approves 79 per cent of submissions, which is up from 48 per cent between 2011 and 2013. A key change in the health technology assessment process has been to give the SMC greater latitude, when assessing medicines, to take into account patients’ lived experience.
On Monday, I announced a further change. From October, defined ultra-orphan medicines—those for the rarest conditions—will be made available on the national health service for at least three years, while information about their results is gathered. The SMC will also have flexibility to allow some orphan medicines to go through the ultra-orphan process when it considers that appropriate. At my request, the Scottish Government’s chief pharmaceutical officer has written to ask the SMC to determine whether Orkambi might be considered in that way.
We have changed the system for individual access to medicines that are not generally available on the NHS. Under the new peer-approved clinical system—PACS tier 2—the cost of a medicine has since 1 June been explicitly excluded from decision-making criteria when a clinician’s request for individual access is considered.
In recent years, we have ensured that the rebate that comes to Scotland as part of the UK’s pharmaceutical price regulation scheme has been invested in access to new medicines. We will ensure that that continues.
First, it is still important to demonstrate clinical effectiveness. We have made funding available through the new medicines fund, which is funded through the PPRS. Anas Sarwar will be aware that that is under negotiation as we speak; I will come back to that.
As for improving the ways to negotiate with drug companies, a number of steps have been taken to better pursue best value for the NHS. NHS Scotland and the Association of the British Pharmaceutical Industry have agreed a new voluntary system to ensure that, for the first time, discounts that are offered to one part of the UK are made available at the same level in Scotland.
We want to go further and help NHS Scotland to negotiate for patients in new ways. Critical to that is ensuring that the new UK PPRS leaves greater scope for NHS Scotland to negotiate with companies about their applications for new medicines. Sadly, the existing PPRS, which expires at the end of the year, places tight constraints on Scotland’s scope for additional negotiation. That prevents opportunities for negotiation, as we cannot contravene the PPRS terms that the UK Government agreed.
We have asked twice for Scotland to be a party to the upcoming PPRS negotiations with the industry, to ensure that we secure the scope for greater flexibility in negotiation, but our requests have been refused. Our aim is to align the implementation of a new negotiation scheme and the implementation of the single national formulary to the outcome of the PPRS negotiations, which we hope will be concluded as quickly as possible. As such, I am happy to accept Miles Briggs’s amendment, as well as the Labour motion. I hope that we can rely on support from members across the Parliament for Scotland to receive a fair deal and the flexibility that we need from the PPRS negotiation.
Today’s motion refers to two specific medicines—one for secondary breast cancer and the other for cystic fibrosis. Just last week, the Parliament heard of the terrible toll that is taken on people who live with cystic fibrosis, and few of us will not have had a family member affected by cancer.
The SMC, NHS Scotland and my officials have been working to help the companies that have developed Orkambi and Perjeta to apply to have their medicines considered flexibly by the SMC. I warmly welcome the undertaking that was offered by Roche to make a new application for Perjeta—that is a positive step. I hope that the makers of Orkambi will submit a fresh application, too, because it is important that clinical effectiveness is established and that is the process that every company must go through. Scottish Government officials met representatives of Vertex Pharmaceuticals this week to discuss its proposals. I hope that it will engage fully and positively with assessments to ensure the clinical effectiveness of its medicines.
The Scottish Parliament has helped to drive forward substantial reforms in this area, but we must also expect that some companies reform some of their practices and come forward with far fairer prices and clear clinical evidence for assessment. Every other pharmaceutical company has to do that.
There is little doubt that decisions around the availability of new medicines are among the most difficult issues that Governments face, which is why the system is independent and not in the hands of politicians. The system has been reformed considerably—any reasonable person would agree with that—which means that we get more drugs more quickly into the hands of more patients. We have made advances and we will not stop in our efforts to make further advances.
I move amendment S5M-12856.3, to insert at end:
“; welcomes the introduction of a new ultra-orphan pathway within the Scottish Medicines Consortium process that will make available, with ongoing evaluation for at least three years, medicines for the rarest conditions; notes the roll-out of the Peer Approved Clinical System in June 2018, replacing individual patient treatment requests, which allows clinicians to seek medicines for their patients that are not currently accepted for routine use by the NHS in Scotland, and which makes clear that the cost of the medicine must not be part of the decision-making process; further notes the commitment of the Scottish Government to continue to use all Pharmaceutical Price Regulation Scheme rebate funding to support access to new medicines; welcomes the commitment of the Association of the British Pharmaceutical Industry for its members to provide Scotland with the same discounts offered elsewhere in the UK for accessing medicines, and believes that pharmaceutical companies should offer NHS Scotland fair prices and should properly engage with health technology assessments in order to demonstrate the clinical effectiveness of their medicines.”
I am pleased to contribute to today’s debate about access to life-prolonging medicines and I thank the Labour Party for bringing it to the chamber. It is an issue of great concern and importance to many patients and their families in Scotland.
Earlier this month, working with Breast Cancer Now, I was pleased to host a summit on access to Perjeta here in Parliament with breast cancer patients, manufacturers, representatives of the Scottish Government and members of all political parties in the Parliament. Attendees at the summit heard moving and powerful testimony from breast cancer patients such as Jen Hardy from Edinburgh, who told us:
“I have lost out on 16 months of precious extra time with my beautiful family because I’ve been denied Perjeta. With every moment that goes by more women are missing out. The drug company, the Scottish Government and the SMC need to keep working together to make Perjeta available on Scotland’s NHS. It’s time to end this injustice.”
I commend Breast Cancer Now and individuals such as Jen and many others for leading such a high-profile and passionate campaign. At the meeting, we heard from another of my constituents, who spoke what were the most beautiful and poignant words that I have heard in the Parliament. The mother of two from Portobello said:
“In my case the differences of the extra time include better mental health reducing the overwhelming guilt at leaving my gorgeous children at such a young age. Instead I can again relax and enjoy time with them. I can also look forward to all the fun bits of being a mum such as being the tooth fairy, being with them as they learn to read and hopefully love books as much as I do, discovering Legoland and all sort of wonderful places and experiences. Perhaps even more important is knowing I will also be there at the difficult times maybe even reassuring them as secondary school and the teenage years approach.”
I hope that those words demonstrate why we are debating this important issue today, and why we must have urgent progress.
Although I welcome Roche’s confirmation that it will make a new bid to the SMC, Scottish campaigners and patients are understandably frustrated and angered by the delays that they face to access Perjeta when it is already available on the NHS in England and Wales.
Similarly, in the case of Orkambi, I was pleased to speak in last week’s members’ business debate that was led by my colleague Maurice Corry, in which I highlighted my constituents’ strong desire for access to the drug, which can, as we heard, transform the quality of life for people with cystic fibrosis.
Parents from around Scotland, including people such as Jenny Landers in my region, are to be congratulated on their campaigning efforts. It is because of them that we are here today and making sure that we achieve this change.
Although today’s debate focuses on Perjeta and Orkambi, I have been contacted in the past few weeks by constituents and families who are campaigning for access for themselves and their loved ones to other specialist drugs that they consider to be absolutely vital. Families of children with rare diseases, such as 5q spinal muscular atrophy types 2 and 3, want the SMC to help provide them with medicines that could radically improve their lives. They desperately want a system that is responsive, transparent and fast.
The Scottish Government, as the organisation that ultimately sets the rules around how the SMC operates, needs to show that it understands and can respond to patients’ wishes. As Opposition MSPs, it is our job to press ministers on this matter and speak up on behalf of our constituents.
Many elements of the Montgomery review are welcome, but there are growing frustrations that they might not go far enough and that the implementation of some of them is taking far too long.
My amendment adds to Anas Sarwar’s motion and reflects what patient groups feel in relation to the need to improve the patient access scheme assessment group. Current processes are failing and they are not able to assess, in the most adequate way, highly innovative medicines. No doubt we will all see a great many more of those come forward in the next few years, as technology advances and genetic profile-specific
drugs emerge into the market. They are going to be for small patient numbers and we must be able provide access to them.
It is clear that too many patients and families across Scotland face barriers to accessing new drugs. The Scottish National Party Government and the Parliament need to make sure that that changes as soon as possible. Sadly, in too many cases that we will hear of today, it is too late for patients and their families. I support Anas Sarwar’s motion.
I move amendment S5M-12856.1, to insert at end:
“, and further calls on the Scottish Government to remove barriers to make access to new drugs easier, including the reform of the Patient Access Scheme Assessment Group.”
We all want patients to be able to access the treatments that they need without delay. It is unthinkable that patients’ health is deteriorating while medicines that could help them are not being used.
It is right that Parliament works to ensure that medicines reach the people who need them. The best long-term solution is to improve the frameworks that surround those decisions, as there will never be time in this chamber to properly consider individual medicines with the urgency that patients and all the organisations and individuals who have campaigned long and hard require and deserve. The amendment that I lodged was clear that all patients need to have confidence that there is a trusted and transparent system for regulating the approval of all new medicines.
Today, Cancer Research UK has emphasised that the SMC plays a vital role in assessing the clinical and cost effectiveness of new medicines independently of the Scottish Government. It is right that there should be an independent process. Ultimately, the efficacy of individual medicines must be evaluated by clinical experts.
I support the motion before us today. Improvements have been needed to our overall frameworks for approving new medicines. The motion rightly highlights that the Government has already committed to improving aspects of negotiating with drug companies.
I acknowledge that we have seen progress towards implementing the recommendations of the Montgomery review over the past year, which the Government’s amendment details. I will support the Government's amendment on that basis, although I appreciate the real concerns that progress has not been fast enough or always clear enough. I also wholly support the position that
“pharmaceutical companies should offer NHS Scotland fair prices and should properly engage with health technology assessments”.
I am glad that the manufacturers of Perjeta are making progress on resubmitting to the SMC, and I implore the manufacturers of Orkambi to do the same. There is no time to be lost.
I will also support the Conservative amendment that proposes that we remove unnecessary barriers to treatment. I am open to some potential reform of the role of the patient access scheme assessment group, although I am not wholly convinced by submissions to the Montgomery review by pharmaceutical companies that said that they want to see the assessment group
“develop their role from gatekeeper to enabler”.
I also have reservations about urging NHS National Services Scotland, the SMC or the Government to move into negotiations that are even less transparent than current processes. Decisions about procurement should always be taken as transparently as possible.
If we are to urge the Scottish Government to take action beyond the SMC process, we cannot rule out other legal routes to procure medicines. The campaign group just treatment wants to see the Scottish Government make use of its powers to pursue a Crown use licence in some cases. I have raised that possibility with the cabinet secretary recently. I appreciate that that might not be a quick solution, but it has the potential to lead to much-needed long-term change on drug pricing.
We cannot ignore the fact that manufacturers have the latitude to change their stance on price. I very much hope that Roche has reached a position that allows the SMC to approve Perjeta for general use, and I encourage Vertex to do the same on Orkambi.
As the just treatment campaign has pointed out, although we cannot alter the efficacy of drugs to make them more cost effective, the price is variable, and the key driver of price will be the patent-backed monopoly that is held by manufacturers. The “Just treatment” campaign has worked with inspiring campaigners such as Dunise MacIver, who have spoken honestly and openly about the difference that accessing Perjeta would make to their care and treatment. Last week, my colleague John Finnie highlighted the experience of his constituent, Hannah McDiarmid, who has grown up with cystic fibrosis and lives with two hours of physiotherapy a day to clear mucus from her chest and lungs.
It is incredible that so many constituents have put so much into campaigning for access to treatment when their own health must be their priority, and they manage really complex treatment regimes. They are inspiring us, but they should be able to focus entirely on their health and wellbeing. We must get access to medicine right in the first place so that no one has to lose time that they could be spending with their families because they are putting their energy into leading campaigns for the treatment that they need.
It is tough to watch and read about breast cancer patients and their ordeal. Who would not want to make policy changes when they learn about Jen Hardy, from Edinburgh, and her HER2-positive secondary breast cancer? She has been denied Perjeta and the 16 months of life that it could give. When someone who is exhausted from the effects of breast cancer considers moving home to get treatment, that shows how much it matters to her. Similarly, the 31-year-old daughter of Jacqueline McEnaney has cystic fibrosis and would benefit from Orkambi. This week, she attended the funeral of the last of her childhood friends with the condition. Orkambi could extend her life and improve her daily quality of life.
Only thanks to advances in medicine is this debate even possible. Previously, there would have been no hope, but now there is. However, with that hope comes a new set of challenges. There is an expectation on the NHS and the state to do everything possible to save our friends and relatives from pain and early death. We want new medicines and innovations to be used by the NHS to improve people’s lives and to encourage greater innovation by industry and researchers. However, that cannot be at any price or effectiveness, because that might have an effect on other treatment and services that are provided by the NHS, which might be equally—if not more—justified.
The SMC process is specifically designed to assess the flow of new medicines from pharmaceutical companies. Because drug discovery is not cheap, that decision involves a difficult set of judgments. It has been recognised that the process for orphan or ultra-orphan conditions requires adjustment, but progress since the Montgomery review has been slow. Even so, the ultra-orphan process has nothing to do with Perjeta. The PACS tier 2 process should give patients a better chance of accessing Perjeta on an individual basis, but why is there a need to use that process when the drug is routinely available in England?
There is a lack of clarity about what is happening with end-of-life drugs such as Perjeta. How can Perjeta be cost effective in England but not in Scotland? Leaving aside the cancer drugs fund, how was a special deal reached with Roche in England but not in Scotland? I hear what the health secretary says about the PPRS, but that does not explain why the Welsh Government has given the go-ahead. Similarly, in Northern Ireland, we are seeing a managed arrangement around the use of Perjeta.
I agree with that approach, but that does not explain why we are in the position that Wales and Northern Ireland seem to be moving ahead, alongside England, while Scotland seems to be incapable of doing so.
Following the rejection last year by the SMC, Roche said:
“unfortunately inflexible pricing rules mean they have been unable to accept our discount in full.”
I do not understand what that means and I would like an explanation from the health secretary when she sums up.
The Government’s amendment says that pharmaceutical companies should offer the same prices across the UK. I agree with that, but that does not explain the situation that Roche is describing. We all want to solve the problem, so we need to have some clarity around that. We know the dilemmas that are involved in trying to get expensive drugs through the system, but we need some clarity and progress for the sake of the patients involved.
It was only a week ago that we had a members’ business debate on access to Orkambi. The Minister for Public Health and Sport’s response then was disappointing and pre-scripted, and it offered little comfort to those for whom that drug could be life saving. I believe that the cabinet secretary has moved on from that today, which is welcome, although I would, of course, encourage her to go further.
The issue is not just access to Orkambi, important though that is, but access to the next generation of drugs to treat cystic fibrosis—the drugs that are being trialled as we speak, which will transform lives, and the drugs that will follow them in a few months, which will treat the underlying causes of cystic fibrosis rather than simply the symptoms. That really will be life changing. According to clinicians, instead of someone with cystic fibrosis dying before they reach 31 years of age, they could live into their 70s or 80s. They would have a normal life expectancy, which is simply extraordinary. We have an opportunity and a duty to do something about that.
Orkambi was licensed for use in 2015. The SMC recognised that it was an important therapy but rejected it on cost grounds in 2016. Two years on from that, Orkambi is available from the drugs company only on compassionate grounds. Meanwhile, people with cystic fibrosis are dying.
Time is something that cystic fibrosis sufferers do not have. A resubmission to the SMC would take six months, and I am not convinced that the appraisal process recognises the contribution to the economy that someone living and working for an additional 30 to 40 years would make. The way in which the SMC measures cost and benefit does not even begin to capture that fully.
I welcome the roll-out of the ultra-orphan medicine pathway. It is great, but the truth is that it applies only to conditions affecting fewer than 100 people. There is a gap in the system in how we treat orphan conditions. Orkambi is appropriate for about 300 cystic fibrosis sufferers, so it does not qualify as an ultra-orphan medicine. It is clear that there is a gap between the ultra-orphan medicine pathway and the SMC process.
I hear that the cabinet secretary is asking for an exemption to be made in the case of Orkambi. That is a start, but it does not address the underlying problem, which is where I would like her to go further. We need a portfolio agreement, not just an agreement about Orkambi. We need an agreement about the next generation of medicines that are coming down the line. I know that that is a new concept that does not fit the processes that the Scottish Government has, but let us not be hidebound by systems if they are not flexible.
Allow me to correct the cabinet secretary as gently as I can. All new treatments in a portfolio agreement have to be licensed first. I would have the same safety concerns as she has, and I would not be recommending that course if I did not think that it was appropriate. At the end of the day, it is about patients.
Here are the countries that have agreed a portfolio deal: Austria, Denmark, Germany, Luxembourg, the Netherlands, Italy, Greece, the United States, the Republic of Ireland and—just this week—Sweden. I ask the cabinet secretary: are all those countries wrong? Do we know better? Are cystic fibrosis sufferers in Scotland different from sufferers in any of those countries?
Jackie Baillie raises an important point. The portfolio approach did include unlicensed medicines the safety of which remained unproven. I am glad that she shares my concerns about that. It is a fundamental problem with the portfolio approach that needs to be addressed.
I am suggesting that we can address it if we have those negotiations. All those other countries have, and negotiations on such an agreement are well under way in England. I understand that agreement will be reached soon—perhaps even before the end of the summer—which will save the lives of cystic fibrosis sufferers in England. I cannot believe that the cabinet secretary wants to send the message today that parents who want to save their children should move to England to do so.
I am grateful that the cabinet secretary is going to meet my constituent Kelli Gallacher next week, but Kelli does not have time to wait: she needs Orkambi now.
As the MSP for Edinburgh Eastern, I have met constituents for whom access to potentially life-altering medicines for themselves or their children is an incredibly pressing concern. The Scottish Government has acted to significantly improve access to medicines in recent years, but I know, from meetings with constituents and the correspondence that I have received from constituents, that accessing certain medicines and treatments has sometimes proved frustrating.
In Scotland, new drugs are appraised in a clear way. The process is independent of ministers and Parliament, and decisions are made by the Scottish Medicines Consortium. The system needs to be fair and consistent, but it also needs to be able to respond swiftly to clinical need. Pharmaceutical companies must play their part in that process by submitting a fair price—ideally, the first time.
The Scottish Government has listened to feedback from patients and has responded to the Montgomery report’s recommendations by implementing a series of reforms to the system and new measures that will make it easier for patients with rare conditions to access new medicines and treatments. It has announced just this week that it has widened the definition of ultra-orphan medicines to include medicines for rare orphan diseases so that patients with rare diseases can get faster access to new medicines and treatments. That means that, if a medicine meets the definition and the SMC considers it to be clinically effective, patients will be able to access the new medicine on the NHS for at least three years while information on its wider effectiveness is gathered.
That follows changes that were made this month that give doctors the right to access licensed treatments that are not generally available on the NHS on a case-by-case basis, making it easier for patients to get access to the specialist medicines that they need. The peer-approved clinical system tier 2 will act as a sort of safety valve in the system for clinicians, and cost effectiveness must not be part of the consideration for non-routine access. That approach is supplemented by the new national appeal panel, and it provides a more flexible pathway for clinicians and their patients.
Those changes reflect the Government’s understanding that more can and should be done in exceptional cases, and they amount to major improvements in the access to new drugs that have the potential to improve the quality of patients’ lives.
The Government’s changes are significant, but it is also vital that pharmaceutical companies play their part by bringing a fair price to the process. As we know, ultra-orphan medicines are expensive, and the SMC’s role is to ensure that the best-value medicines are available to the NHS in Scotland. I understand that, following encouragement from the Scottish Government, Vertex Pharmaceuticals and Roche have submitted new applications to NHS National Services Scotland. I hope that that results in agreement being reached on fair prices to enable patients in Scotland to access the medicines.
I hope that those recent announcements go some way towards reassuring my constituents and patients across Scotland that the system is being reformed and taken seriously and that access to the latest medicines for those who need them is being significantly improved.
I thank the Labour Party for bringing an extremely important topic to the chamber for debate.
I ask members to imagine a situation in which a family member or loved one could live longer if only they were given access to a medicine that they knew already existed, and then to imagine their frustration should that person be denied that drug. By so doing, we get to the crux of why the debate is so important.
We have seen in the media the personal testimonies of patients who need the drugs in question. Last month, a young woman wrote to the First Minister to beg for access to Orkambi. That drug would improve her chances of living beyond the age of 31. A few weeks ago, as part of a Breast Cancer Now campaign, I met a campaigner who desperately wants and needs Perjeta in order to increase the time that she has left.
The specialist drugs Orkambi and Perjeta are potentially life changing. Orkambi is a precision medicine that targets the root cause of cystic fibrosis and has the potential to improve the lives of more than 336 people in Scotland by preserving and restoring full lung function. Perjeta is a drug for people with HER2-positive breast cancer that is said to prolong the lives of women with incurable breast cancer by up to 16 months.
That is why it is so important to discuss those drugs’ availability in Scotland. The Scottish Conservative Party has consistently called for both drugs to be available on the NHS. Miles Briggs hosted cross-party talks on the availability of Perjeta this month, and in May Ruth Davidson raised the issue at First Minister’s question time and stated that breast cancer sufferers had travelled to England to access the drug. Last week, Maurice Corry led a members’ business debate and called for Orkambi to be made available in Scotland.
Cost will always be a factor in making such decisions, of course, but it is clear that the Scottish Government must provide clarity about what it intends to do. There are underlying issues and reform is needed. With regard to negotiations, the SMC of course makes decisions independent of Government, but it is, after all, the Government that sets the framework under which those decisions are made. That is why the Scottish Government must prioritise putting in place a negotiating system that will ensure greater access to the drugs. It is 18 months since the Scottish Government promised to do so, so I call on the cabinet secretary to provide a clear deadline for when we can expect that. Furthermore, as is alluded to in Miles Briggs’s amendment, we must push for reform of the patient access scheme assessment group to make access to high-cost drugs easier for patients.
In addition to those reforms, I echo the calls that are being made by Miles Briggs for a cross-border arrangement to ensure that no one misses out on crucial care. Medicines are also available in Scotland that are not available in England, which is why it is so vital for us to work together and share resources.
I reiterate my support for the debate. The time has come for greater clarity surrounding new medicines. We have seen in recent weeks just how pertinent are demands for life-changing drugs to be made available in our NHS. Our patients suffer as a result of lack of decision making and complex discussions around cost—which must, of course, be factored in. However, I call on the Scottish Government to put in place urgently a new system of negotiation for such life-saving drugs. Only then will patients get the chance that they deserve to extend their lives and give relatives a source of comfort.
I commend my colleague and friend Anas Sarwar for his persistent focus on access to medicines, which has allowed us to devote Labour’s debating time to the issue today.
I will share my experience of supporting constituents who have cancer, and I want to make three points: about the wider situation that faces the NHS; about the Montgomery review—in particular, the replacement for individual patient treatment requests; and about Jen Hardy and her battle for Perjeta.
I inform the Cabinet Secretary for Health and Sport that a woman came to my surgery on Friday to see me about a family member who had waited more than a year for an endoscopy from NHS Lothian. She never got the treatment that she needed and died from stomach cancer earlier this year. Today’s debate about access to medicine has to be seen in the wider context of the pressures on our NHS—in particular, the Government’s consistent failure to deliver on treatment time guarantees, and the fact that cancer is no exception to that record of failure.
I am pleased that the Government has accepted the Montgomery review recommendations to replace IPTRs with the new PACS tier 2 system, as Anas Sarwar mentioned. I have direct experience of trying to support two constituents in the IPTR process: one was successful and one was not. The first was a woman who walked into a constituency surgery a few years ago who needed help to fill out the paperwork for IPTR to access the drug Kadcyla for breast cancer. She was ultimately successful, but I believe that that was only because of the sheer force and pressure that was put behind the campaign by Breast Cancer Now, which did formidable work in fighting for that drug.
The second constituent whom I tried to support in an IPTR was a woman who had bowel cancer. One of the hardest things that I have ever had to do as an MSP was visit her in her house in Edinburgh and have a conversation about why she could not get the drug that she needed after she had devoted her entire career to the NHS. She was a paediatric nurse: every waking moment of her working life had been spent in the national health service, but I was sitting in her living room trying to explain why she could not get the drug that would have saved her life. Sadly, she passed away in February this year, having never had the treatment that she needed.
We have heard from Miles Briggs and others about the situation that is facing Jen Hardy. She should be spending her final months watching her daughter graduate and get married, and enjoying Christmas with her family, but she has been spending her time standing outside Parliament educating MSPs on a drug that would have given her 16 more months of life. The Government has been dragging its heels for a long time over Perjeta. In fact, we have been waiting longer than that drug would give Jen Hardy in extra months to spend with the people who desperately want her by their side.
If the cabinet secretary takes away one thing from the debate, it should be that she should, please, stop dragging her heels, because there are people who need the drugs now. Every member will have experienced trying to support a cancer patient through one of the most difficult experiences of their lives and who is being hit by the system time and again. The system is failing such patients and we could do much more to improve it. The power to do that lies in the health secretary’s hands. I hope that she steps up and uses it.
I refer members to my entry in the register of interests, which shows that I am a registered mental health nurse and that I currently hold an honorary contract with NHS Greater Glasgow and Clyde.
Like other members who are present, I know constituents and friends who have either had or been affected by breast cancer or cystic fibrosis. Those illnesses impact not only on the patient, but on the families and friends who support them. As many members across the chamber have, I have heard heartbreaking stories from constituents whose lives have been turned upside down by breast cancer and cystic fibrosis. For that reason, I fully applaud the tenacious campaigns that are being led by Breast Cancer Now and the Cystic Fibrosis Trust calling on authorities to widen access to medicines for such conditions. Those organisations’ campaigning has helped to educate MSPs and the wider public on the merits of widening access to the drugs. For that we owe them a debt of gratitude.
As we have heard, the Scottish Government has in recent years significantly improved access to new medicines. Figures show that between 2011 and 2013, the combined acceptance rate for orphan and cancer medicines was 48 per cent, whereas in the past three years, under the new approach, the Scottish Medicines Consortium has approved 79 per cent of such medicines. There can be no doubt that those drugs have changed lives.
However, we can always improve and build on our processes and learn from our experiences and from evidence-based best practice from other nations. I welcome the Government’s commitment, following the recommendations that were laid out in the Montgomery report, to reform the systems that are currently in place and to introduce changes that will enable medicines to get to the people who need them. As we have heard, the Cabinet Secretary for Health and Sport announced only yesterday that the Scottish Government is introducing a new definition of ultra-orphan medicines, which will give the Scottish Medicines Consortium the ability to treat some medicines for rare orphan diseases as ultra-orphan medicines. In effect, the changes will mean that if a medicine meets the new definition of an ultra-orphan medicine and the SMC considers it to be clinically effective, it will be made available on the NHS for at least three years while information on its effectiveness is gathered.
That is one of a number of steps that are being taken to ensure that access to vital medicines is widened. With those new rules for medicines, faster access to new treatments will become a reality.
I wish to reiterate that medicine approval decisions are not taken by MSPs or the Government. That is the role of the Scottish Medicines Consortium, which, as the cabinet secretary has rightly said, acts independent of ministers and Parliament. Nobody wants to be in a situation in which certain medicines are rejected, but it is entirely appropriate that such decisions are taken carefully, based on clinical evidence, and made by an independent body.
As others have done, I welcome Roche’s announcement that it is to make a new submission to the SMC on Perjeta. I urge Vertex to do likewise for Orkambi as quickly as possible. However, we cannot allow our health service to be held to ransom by pharmaceutical companies, so we must encourage them to offer fair and transparent prices for their products. Everyone here agrees that we want such drugs to be made available to the people of Scotland, but there must be fairness in the cost of the drugs that are supplied. I therefore welcome the commitment that has been made by the Association of the British Pharmaceutical Industry that its members will provide Scotland with the same discounts that are offered elsewhere in the UK for accessing medications.
Today, Parliament has spoken with one clear voice, calling all pharmaceutical companies to play their part and to bring a fair price to drug appraisal processes the first time. It is quite right that people should not lose out to profits.
I am delighted to have the opportunity of taking part in today’s debate.
The serious issues of cystic fibrosis and breast cancer are two completely separate conditions, but they have a common thread, in that they are both attributed t o genetics. As we have seen, individuals who have those diseases have tried their best to do all that they can, and companies have consistently had an uphill struggle to ensure that they can successfully provide treatments for patients.
However, thankfully—and due in no small part to current knowledge and expertise—two drugs are now available for cystic fibrosis and HER2-positive breast cancer.
First, Orkambi is different from traditional treatments for cystic fibrosis because it is a precision medicine. With traditional medicines, damage occurs and patients have seen their illness progress. Precision medicine targets the root cause and has the potential to ensure that lung function is restored and that the patient’s decline subsides. The Cystic Fibrosis Trust has recognised that 336 people in Scotland could currently benefit from having access to that drug; that is one third of the 900 people across the country who live with CF.
P erjeta is a newly developed drug for women with HER2-positive breast cancer, and has been created by the pharmaceutical company Roche. Today, we have heard how women in England and Wales can get the drug, but those in Scotland cannot. It gives patients with cancer the opportunity to have their treatment increased for 16 months, which is a lifeline for many and ensures that they spend more time with their families and loved ones. They need access to it now—not later.
We know the facts about Orkambi and Perjeta, and their benefits, but they are not available to Scottish patients, who cannot understand why they are not being seen as a priority. Why are they not being given those opportunities? The life expectancies of many are shortened, and they die because they do not have the drugs.
The Scottish Conservatives have made it quite clear, on numerous occasions in this Parliament, that Orkambi should be available—we have been discussing it since 2016. Indeed, in a debate last week we called for it to be made available. I pay tribute to Maurice Corry, who called for a portfolio approach whereby medicines for cystic fibrosis could become available for patients when they are manufactured and licensed.
Deals of that type have already taken place in other countries, and people want to know why cost and bureaucracy are stopping them from happening here. Doctors can move patients on to new medicines if they believe that their access to them might ensure that they have longer life expectancies, and we should provide that. Only last month, Ruth Davidson spoke about Perjeta at First Minister’s question time. I also pay tribute to my colleague Miles Briggs, who last month held cross-party talks on the topic.
We have also talked in the Scottish Parliament about having a cross-border arrangement for Perjeta, to ensure that no one misses out on that crucial drug.
It all comes back to choice. The Scottish Government is making the choices. We must make sure that those are the right choices for the people of Scotland. They deserve nothing less. Enough is enough.
I welcome the opportunity to take part in this debate on access to new medicines. The member who lodged the motion and all of us in the chamber want to achieve the best results for all sufferers of rare conditions who require orphan, ultra-orphan and end-of-life medication.
For the sufferers and their families, the importance of the issue cannot be overstated. I join other members in paying tribute to those who campaign tirelessly on the issue. As MSPs, we all have cases where access to new medicines would transform the lives of individuals, so it is critical to ensure that robust, independent processes are in place to bring into use new drugs.
The Montgomery review commissioned by the Scottish Government to look into the issue made a number of recommendations, including on datasets, definitions, negotiations, new ultra-orphan medicines pathways and arrangements on funding, which the Government has confirmed that it will be implementing.
The Montgomery review concludes that access to end-of-life, orphan and ultra-orphan medicines has increased through steps taken by the Government, including the use of individual patient treatment requests and peer approved clinical system packs. Indeed, the percentage of new drugs approved has increased from 48 per cent in the period in 2011 to 2013 to 79 per cent over the past three years as a result of investment and reforms to approval processes.
The Scottish Government has put in place the new medicines fund to provide additional support to NHS boards to meet the costs of the drugs, and it commits to continuing to use all the funding from the pharmaceutical price regulation scheme rebate to support the NMF.
The Scottish Medicines Consortium will introduce a new ultra-orphan medicines pathway, with an option to recommend a medicine on an interim basis.
Everything needs to be looked at to make sure that there are no gaps. I have been outlining the changes that the Government has made. What it has done and what it is looking at will go some way to address that issue and should continue to do so.
With the roll-out of PACS, doctors can, on behalf of patients, seek access to licensed treatments not generally available on the NHS. A new national appeals panel will also be introduced for individual requests to allow for medicines not approved by the SMC. In addition, greater cognisance of lived experience will be taken into account in decisions to fund new medicines.
The issue of negotiation figures prominently in the Labour motion, which
“calls for a new system of negotiation to be implemented”.
The market for medicines, particularly new medicines, is complex. It involves assessments of the recovery of research and development costs, which are essential to ensure that the pipeline of new medicine development is not slowed.
Matters are further complicated by the multilayered processes involved in UK pricing negotiations. The Scottish Government’s calls for the UK’s PPRS to provide full transparency on pricing among the four UK Administrations, to ensure that NHS Scotland is fully engaged in the process and can leverage best pricing as a consequence, is to be welcomed. Given that ultra-orphan drugs are often expensive, it is vital that pharmaceutical companies play their part and bring a fair price first time to the process.
Developments in drug technology will continue apace. That is to be welcomed, because developments in that area have provided cures to conditions that were, until recently, impossible to treat. The needs of sufferers who require access to the latest medicines is a priority and it is critical to ensure that the process in place for approvals delivers for them. Therefore, I welcome the Scottish Government’s continuing steps to improve access to new medicines and its calls for all parties to get around the table to make progress on the specifics.
I am pleased to close the debate on behalf of the Scottish Conservative Party, and I thank the Labour Party for giving us the opportunity to highlight once again what amounts to an anomaly in the process of approving drugs for general use, particularly portfolio medicines. That is especially true in the cases of the drugs Perjeta and Orkambi.
We have heard how Perjeta can prolong the lives of women who are suffering from terminal breast cancer by up to 16 months, and we have heard how Orkambi, unlike traditional medicines for the treatment of cystic fibrosis, targets the root cause of the condition and has the potential to preserve or even restore lung function, thereby improving life expectancy and the quality of life of patients.
In speeches to the chamber, I tend to shy away from quoting constituents, but in this case I will make an exception, because their experiences and words highlight the issue that we are debating far better than I ever could. Willie Rennie has mentioned Ruth McEnaney, who has a young son. Ruth’s mother wrote to me and asked that I speak on her behalf. She says:
“From this early age her daily regime of medication was huge, 60 tablets a day with added ones when required and intravenous antibiotics on occasion too. She has also had daily physiotherapy from us twice a day and every kind of activity added to keep her fit and active, keeping her lungs in good shape. This must have cost us thousands of pounds over the years, with dancing 4/5 times a week, singing and trumpet lessons etc. I have never regretted one penny of what we have spent on this wonderful, kind hearted intelligent young woman. Having completed a degree in performance music, she has gone on to work with children and young adults with Autism—now a piano teacher herself. She also has worked extremely hard on her fitness as an adult which is the main reason she has got to being 31 years old!”
The age of 31 has already been mentioned as a key age.
“As I said before she is now a Mum too, dedicating her energy to bringing up her 4 year old son, but now on a downward spiral with her health, having more hospital admissions in the last 6 months than ever before and struggling with her lungs and her bowel with blockages now a regular occurrence and chest infections too.”
Ruth’s mother goes on to say:
“Ruth is desperate to stay alive to see her son grow up, to take him to school, high school and beyond, which is what most of us parents take for granted. I cannot therefore understand why there is a drug which could potentially transform her life, sitting on a shelf, while people say the cost is not worth it for her and many more wonderful, courageous young cystic fibrosis warriors. They have battled for so long already along with us, I’m hoping and praying that today will be a landmark day, to change their lives forever, giving them the gift of improved health and long life!”
As Willie Rennie said, today, Ruth attended the funeral of her last surviving friend from her days at the cystic fibrosis clinic at Crosshouse hospital. He was 26 years old—five years younger than my constituent’s daughter. Time is precious when we are talking about improving the quality of people’s lives, giving them time with their families and allowing them to continue to achieve, as Ruth has done.
Most debates in this place are about finding a way to gain a political inch on our opponents, to dodge the bullet of an intervention or to get out of here unscathed having landed a blow, but this afternoon’s debate is not. It is not about politics. It is about finding a way—and doing so quickly—to resolve a situation that is eminently solvable.
I fully recognise that it is not for politicians to make medical decisions and that there is always a tension between those medicines that are approved and those that are rejected, but I make a plea to the cabinet secretary to get the parties round a table and find a solution, because that is most definitely within her power.
It has been a good debate, in which we have heard some very powerful speeches. I hope that it has united the chamber in a number of respects; I will come back to that shortly.
I want to deal with as many of the issues that have been raised as possible, although, obviously, I will not be able to deal with all of them in the time allotted. Annie Wells made a point that addressed the point that was made by Anas Sarwar and others: of course there will be drugs that are not available either north or south of the border, because we have different assessment processes. There will be drugs available in Scotland that are not available in England. That is the nature of the systems that we have, but they should all be based on the clinical evidence. I will come back to that, because I thought that Jackie Baillie made an important point in that respect.
Anas Sarwar, Jackie Baillie and others talked about the portfolio approach. The portfolio approach was not raised by Montgomery or anyone else in their recommendations. Officials in both Scotland and England all have concerns about it for two very important reasons. One is that it seeks to bypass the standard health technology assessment process. I have not heard anyone in the chamber disagree that clinical effectiveness has to be tested and established; everyone has agreed that it does and said that the medicines have to be licensed. Therefore, the manufacturers of Orkambi need to revisit their current portfolio approach.
That is a very powerful message for the chamber to send to Vertex and other pharmaceutical companies. I agree that the systems have to be reformed, but clinical effectiveness still has to be established and the medicines have to be licensed for patient safety reasons. That is something that Jackie Baillie said, too.
I did. Let me quote from a Vertex statement that was made following its meeting with the Scottish Government on 18 June. Vertex says that it will accelerate
“access to our pipeline of potential new CF treatments after license”.
The tests of the clinical safety of that drug that the cabinet secretary and I want to see will have been carried out. There is nothing to stop the cabinet secretary from engaging in a portfolio agreement that would save lives.
It is not for the Government to do that—it is for the SMC. However, there were unlicensed medicines in that portfolio. If things have moved on, that is to be welcomed. Dialogue continues, of course, and there has been a lot of dialogue between Vertex and Scottish Government officials and also national procurement. From the last round of discussions, I hope that progress can be made and I certainly encourage that.
Willie Rennie raised an issue, and I want to be as clear as I can be in my response. He asked why the Perjeta deal was not available in Scotland. NHS England has come to a confidential commercial deal with Roche, the details of which cannot be shared between Administrations because of that commercial confidentiality. That is unlike the situation for standard discounts, whose details can be shared. That is why we need transparency and price parity written into the new PPRS scheme. I cannot emphasise that enough; it is very important. The lack of transparency prevents me from telling Willie Rennie some of the detail that might be quite helpful.
I will write to members about the specific issues that they raised, but the message that we can all agree on in this debate is that we want medicines to get into the hands of patients as quickly as possible. I want that but, rightly, those decisions are not made by politicians. However, we have improved the systems in order to deliver that aim. I encourage Vertex to continue with those discussions, to get a new submission to the SMC and to make sure that the medicine is available at a fair price as quickly as possible. That would be well received.
Thank you, Presiding Officer. This has been a productive debate with passionate and well-informed contributions from across the chamber. At one level, discussions on the licensing of medicines and negotiations with drug companies can become quite technical and abstract. However, let us never forget that the outcomes of such debates have reaI-life impacts for the individuals affected. We had passionate contributions from Anas Sarwar, Jackie Baillie, Kez Dugdale, Miles Briggs, Ash Denham and Brian Whittle.
Orphan and ultra-orphan medicines may treat only a few hundred people per year in Scotland, but that does not mean that they should be overlooked. Every delay to improving the system of negotiations means another day less for women with incurable HER2-positive breast cancer to spend with their loved ones, and another day in which lives are lost because people with cystic fibrosis are denied access to the drug Orkambi.
This week, the Government has made some improvements regarding access to medicines with the PACS tier 2 system and the newly announced ultra-orphan pathway—steps in the right direction, but action is still too slow. It has been two years since the Montgomery review, and many patients still do not have access to those important life-sustaining drugs. Campaigners are rightly frustrated at the lack of progress that has been made in Scotland in negotiations with the manufacturers Roche and Vertex about Perjeta and Orkambi. That is especially the case when a deal has been made to allow women access to Perjeta through the NHS in England and Wales—the discrepancy in provision just a few miles across the border is a daily, growing injustice.
As my colleague Anas Sarwar said in his excellent speech:
“Women in England, Wales and Northern Ireland can get Perjeta on the national health service as a matter of course, but women in Scotland cannot get Perjeta. The drug has been rejected three times by the Scottish Medicines Consortium as it is not considered cost effective, despite it being recognised as clinically effective. I ask again: what cost life?”
In another excellent speech, Miles Briggs talked about the summit that he hosted on Perjeta, and I welcome the work that he has done on that. He said that the message from campaigners is to keep on working with the Scottish Government, the SMC and the industry to stop this injustice, and pointed out that of course patients value the extra few months with their families and young children, but that too many families face barriers to getting drugs.
Alison Johnstone made some excellent points, but I highlight her point about the Government getting a Crown use licence, which is a very important initiative, and the fact that we have many patent-backed monopolies. She said that it is vitally important to change the approval frameworks.
Willie Rennie made some excellent points as well. He talked about advances in medical science, which now give hope to many people who in the past would have had very little hope. He said that of course we need innovation in health but that it cannot be
“at any price or effectiveness”.
Jackie Baillie made a very powerful and personal speech about examples that she has come across. She said that there is an opportunity now to do something about the issue and mentioned the portfolio deals that many countries such as Sweden and the USA have carried out.
Ash Denham talked about her constituents with life-threatening conditions and their concerns about pricing and said that companies need to play their part by offering a fair price. Many other speakers, including Annie Wells, Ivan McKee, Clare Haughey and Alexander Stewart, made excellent speeches.
I see that I have very little time left, Presiding Officer. Medical research is growing and developing apace. If our systems for approval are too slow in response—if we continue to be reactive instead of proactive—the same problems will occur again and again as new drugs are developed and medical treatment moves forward.
New pathways and systems may sound good but they will not have the confidence of patients, or of members in this chamber, unless they are shown to achieve real results. I therefore urge the Government to heed the campaigners’ calls and act now without delay to make Perjeta and Orkambi available on the NHS to those who need them now. Only then, and then only, can it be claimed that true progress has been achieved.